Kevin J. Coote

This paper develops lipid nanoparticles to deliver CRISPR/Cas9 for site-specific CFTR gene integration in CF airway cells, achieving 3-3.5% gene editing and 80% Cl- current restoration.

The paper demonstrates that ACE-tRNAs can suppress nonsense mutations in CF by rescuing CFTR transcript abundance and channel function in multiple models.

This study develops an iPSC platform to test CFTR modulators in cystic fibrosis patients with rare mutations, showing genotype-specific responses to drugs.